Subsequent studies by other investigators confirmed those early results, ultimately leading to the FDA approval of ...

Since the approval of Luxturna, there has been a wave of gene therapies advancing in clinical trials to treat genetic vision ...

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their ...

Panelists discuss how emerging intrathecal gene therapy for older patients with spinal muscular atrophy (SMA) shows modest ...

The financing will support the ongoing interventional Phase I/II ASTRA trial and the observational POLARIS study of SB-007.

In the first part of this series, we explored how early genetic screening and gene therapy transform the lives of newborns ...

Glioblastoma has been deadly and hard to treat, but researchers say they’ve made progress with bivalent CAR T-cell therapy ...

Oregon Health Plan (OHP) members will see increased access to lower-cost, lifesaving cell and gene therapies. The state is ...

Leaders from the world of cell and gene therapy shared their personal stories and policy recommendations in an extraordinary ...

A Falmouth teen, Jayden Wilsey, is the first to receive gene therapy to treat sickle cell at Boston Children's Hospital.